A Study of RG-012 in Subjects With Alport Syndrome

Genzyme

Status and phase: Completed; Phase 1

Conditions: Alport Syndrome

Treatments: Drug: RG012

Study type: Interventional

Funder types: Industry

Identifiers

NCT03373786

PDY16327

RG012-06 (Other Identifier)

Details and patient eligibility

About

This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome.

Full description

This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.

Enrollment: 4 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Males or females, ages 18 to 65 years
2. Confirmed diagnosis of Alport syndrome
3. eGFR between 40 and 90 mL/min/1.73m2
4. Proteinuria of at least 300 mg protein/g creatinine
5. For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
6. Willing to comply with contraception requirements

Exclusion criteria

1. Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
2. End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
3. Any other condition that may pose a risk to the subject's safety and well-being
4. Female subjects who are pregnant or lactating

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

---

4 participants in 2 patient groups

RG-012 Single Dose

Experimental group

Description:

1.5 mg/kg RG012 subcutaneous injection

Treatment:

Drug: RG012

RG012 Every Other Week

Experimental group

Description:

1.5 mg/kg RG012 subcutaneous injections every other week

Treatment:

Drug: RG012